HOOKIPA Pharma Reports Second Quarter 2019 Financial Results and Clinical Progress Highlights

On August 12, 2019 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics, targeting infectious diseases and cancers based on its proprietary arenavirus platform, reported recent clinical progress highlights and financial results for the second quarter ended June 30, 2019 (Press release, Hookipa Biotech, AUG 12, 2019, View Source [SID1234538622]).

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"In the second quarter of 2019, HOOKIPA achieved a number of major milestones, including clearance to initiate our first clinical trial in immuno-oncology and acceptance by Gilead of a set of 10 HBV viral vectors for further testing," stated Joern Aldag, HOOKIPA’s Chief Executive Officer. "With the FDA’s clearance of our IND application for HB-201, we are advancing our oncology program into clinical development, and are also aiming to demonstrate that our technology can effectively super-charge the natural defense mechanisms in humans and deliver prevention or cure for the benefit of seriously ill patients."

R&D Pipeline Update and Clinical Progress

HB-101, a prophylactic vaccine for cytomegalovirus
HOOKIPA’s lead product candidate in infectious diseases, HB-101, is in a Phase 2 clinical trial in cytomegalovirus-negative patients awaiting kidney transplantation from living cytomegalovirus-positive donors. The majority of sites have been activated and HOOKIPA expects safety and immunogenicity data from the first cohorts enrolled in the first half of 2020, with preliminary efficacy data to follow in the second half of 2020.

HB-201 and HB-202, a program for the treatment of HPV associated cancers
In July 2019, HOOKIPA announced that its Investigational New Drug (IND) Application for a Phase 1/2 clinical trial of HB-201, a TheraT-based immunotherapy for the treatment of Human Papilloma Virus (HPV)-positive cancers, became effective following the clearance by the U.S. Food and Drug Administration (FDA). HOOKIPA plans to initiate a Phase 1/2 clinical trial of HB-201 in patients with treatment-refractory HPV16+ cancers in the second half of 2019. This will be HOOKIPA’s first clinical trial in immuno-oncology.

In addition, HOOKIPA intends to file an IND application with the FDA for HB-202 in the first half of 2020, and to commence a Phase 1/2 trial combining HB-201 and HB-202, both with and without a checkpoint inhibitor, in patients with treatment-refractory HPV16+ cancers in late 2020.

Strategic collaborations

Progress under Gilead collaboration for therapeutic hepatitis B virus (HBV) and human immunodeficiency virus (HIV)
In May 2019, HOOKIPA achieved a $2m research milestone for HBV by designing and delivering 10 research-grade vectors to Gilead Sciences, Inc., or Gilead, along with the characterization of these vectors and delivery of a data package for the HBV program. These research vectors will be subject to further pre-clinical testing in order to validate a clinical candidate for novel combination therapies for the treatment of HBV. This follows the delivery of 14 research-grade vectors for the HIV program in January 2019.

Board and management

David Kaufman joined HOOKIPA’s Board of Directors
In April 2019, HOOKIPA announced the appointment of David R. Kaufman, M.D., Ph.D., to its Board of Directors. Dr. Kaufman currently serves as Chief Medical Officer of The Bill & Melinda Gates Medical Research Institute. Dr. Kaufman’s expertise as an immunologist and in oncology research and development are expected to be a tremendous addition to help maximize the potential of HOOKIPA’s proprietary arenavirus platform to target infectious diseases and cancers.

Second Quarter 2019 Financial Results

HOOKIPA’s net loss for the three months ended June 30, 2019 was $12.1 million, compared to a net loss of $5.8 million for the three months ended June 30, 2018.

Revenue was $4.1 million for the three months ended June 30, 2019, compared to $0.6 million for the three months ended June 30, 2018. The increase was due to recognition of revenue under the Collaboration Agreement with Gilead.

HOOKIPA’s research and development expenses for the three months ended June 30, 2019, were $13.9 million, compared to $6.2 million for the three months ended June 30, 2018. The primary driver of the increase was an increase in direct research and development expenses of $6.4 million. Direct research and development expenses increased primarily due to the preparation costs of clinical trials for HOOKIPA’s HB-201 and HB-202 programs and the expansion of earlier stage programs. In addition, costs related to HOOKIPA’s collaboration with Gilead contributed to the increase in direct expenses. Internal research and development expenses increased by $1.3 million, primarily as a result of increased research and development headcount.

General and administrative expenses for the three months ended June 30, 2019 were $3.8 million, compared to $1.4 million for the three months ended June 30, 2018. The increase was mainly due to the growth in headcount in HOOKIPA’s general and administrative functions and an increase in professional and consulting fees as well as costs associated with ongoing business activities and costs to operate as a public company.

HOOKIPA’s cash and cash equivalents as of June 30, 2019 were $135.2 million compared to $48.6 million as of December 31, 2018. The increase was primarily attributable to $37.3 million in net proceeds received from the issuance of shares of Series D convertible preferred stock in February 2019, and $74.6 million in net proceeds received from

HOOKIPA’s initial public offering in April 2019, offset by cash used in operating and investing activities. On April 23, 2019, HOOKIPA completed an initial public offering of its common stock by issuing 6.0 million shares of its common stock, at $14.00 per share.

Upcoming Investor Events

· Wells Fargo 2019 Healthcare Conference, September 4 – 5, 2019

· BioCentury Conference NewsMakers in the Biotech Industry, September 6, 2019

· Bank of America Merrill Lynch Global Healthcare Conference, September 18-20, 2019

Equillium Reports Second Quarter 2019 Financial Results and Recent Highlights

On August 12, 2019 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging deep understanding of immunobiology to develop products to treat severe autoimmune and inflammatory disorders with high unmet medical need, reported financial results for the second quarter 2019, and recent business highlights (Press release, Equillium, AUG 12, 2019, View Source [SID1234538621]).

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"Since our last quarterly update, we took significant steps forward in the clinical development of our lead therapeutic candidate, itolizumab, with the initiation of the Phase 1b EQUIP proof-of-concept trial for the treatment of uncontrolled asthma, and acceptance of our IND application by the FDA for the treatment of lupus nephritis," stated Daniel Bradbury, chairman and chief executive officer of Equillium. "With two clinical trials now up and running, and a third trial in lupus nephritis planned to commence later this year, we are well positioned to establish the broad clinical utility of itolizumab on our path toward helping improve the lives of patients with severe immuno-inflammatory disorders. We look forward to several important clinical milestones through the end of 2020."

Business Highlights:

Initiated the EQUIP Phase 1b proof-of-concept trial evaluating itolizumab for the treatment of uncontrolled moderate to severe asthma

IND application accepted by the FDA for a Phase 1b proof-of-concept trial of itolizumab for the treatment of lupus nephritis

Continued to advance the Phase 1b portion of the EQUATE trial evaluating itolizumab for the frontline treatment of acute graft-versus-host disease (aGVHD)

Expanded Scientific and Clinical Advisory Team with the appointments of Tom Daniel, M.D., Brian Kotzin, M.D. and Larry Steinman, M.D.

Upcoming Milestones:

Planned initiation of the EQUALISE trial – a Phase 1b proof-of-concept trial of itolizumab for the treatment of lupus nephritis during the second half of 2019

Data from the Phase 1b portion of the EQUATE aGVHD trial expected during the first quarter of 2020

Data from the EQUIP Phase 1b proof-of-concept trial of itolizumab for the treatment of uncontrolled moderate to severe asthma expected in the second half of 2020

Second Quarter 2019 Financial Results

Research and development (R&D) expenses. Total R&D expenses for the three months ended June 30, 2019 were $4.3 million, compared with $0.5 million for the same period in 2018. The increase in R&D expenses was primarily driven by additional costs related to regulatory and clinical development activities associated with the EQUATE, EQUIP and EQUALISE clinical trials, increased headcount expenses, and preclinical research activities to support Equillium’s clinical development program.

General and administrative (G&A) expenses. Total G&A expenses for the three months ended June 30, 2019 were $2.2 million, compared with $0.6 million for the same period in 2018. The increase in G&A expenses was primarily driven by additional costs related to increased headcount expenses, costs related to being a public company and legal and professional fees.

Net loss. Net loss for the three months ended June 30, 2019 was $6.1 million, or $0.35 per common share (basic and diluted), compared with a net loss of approximately $1.8 million, or $0.16 per common share (basic and diluted), for the same period in 2018.

Cash and cash equivalents. As of June 30, 2019, Equillium reported total cash, cash equivalents and short-term investments of $56.9 million, compared to $65.9 million as of December 31, 2018.

Applied Therapeutics Reports Second Quarter 2019 Financial Results

On August 12, 2019 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing novel drug candidates in indications of high unmet medical need, reported financial results for the second quarter ended June 30, 2019 (Press release, Applied Therapeutics, AUG 12, 2019, View Source [SID1234538620]).

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"During the second quarter, we continued to execute on advancing our robust pipeline of novel drug candidates through the clinic," said Shoshana Shendelman, Founder, Chief Executive Officer and Chair of the Board of Applied Therapeutics. "We initiated our Phase 1/2 trial of AT-007 in Galactosemia in June and reported favorable Single Ascending Dose data from healthy volunteers last week. We look forward to advancing AT-007 through the next portion of the trial, which includes treatment of adults with Galactosemia. In addition, we are preparing for the dosing of the first patient in our registrational Phase 3 trial for our lead asset, AT-001, in Diabetic Cardiomyopathy (DbCM), which we expect to occur in the third quarter."

Recent Highlights

Reported Single Ascending Dose Data from Healthy Volunteer Portion of Phase 1/2 ACTION-Galactosemia Trial Evaluating AT-007. In August 2019, we announced the completion of the Single Ascending Dose (SAD) healthy volunteer portion of the Phase 1/2 study of AT-007 in Galactosemia. AT-007 was well tolerated, with no drug-related adverse events or dose-limiting toxicities reported. The study, referred to as ACTION-Galactosemia, was initiated in June 2019 and is designed to investigate the safety and pharmacokinetics (PK) of AT-007, a central nervous system (CNS) penetrant Aldose Reductase (AR) inhibitor in healthy volunteers, and biomarker effects in adult subjects with Galactosemia. Data from the adult Galactosemia patient portion of the trial is expected in the fourth quarter of 2019. We plan to employ recent FDA guidance permitting biomarker-based development in low prevalence, slowly progressing rare metabolic diseases, such as Galactosemia.

·Presented Phase 1/2 Data Highlighting Safety and Efficacy for AT-001 in DbCM at the American Diabetes Association (ADA) 79th Annual Scientific Sessions in San Francisco. In June 2019, we presented Phase 1/2 Data Highlighting Safety and Efficacy for AT-001 in DbCM at the ADA Annual Scientific Sessions. The data, presented as part of the Late Breaking session, demonstrated that AT-001 was well tolerated at all dose levels, and target engagement was confirmed by potent AR inhibition as evidenced by significant reductions in sorbitol, a pharmacodynamic biomarker of AR activity. AT-001 also improved selectivity and affinity for AR and resulted in potent AR inhibition.

·Received FDA Orphan Drug Designation for AT-007 in Galactosemia. In May 2019, we received orphan drug designation for AT-007 in Galactosemia. The designation allows Applied Therapeutics to qualify for a number of incentives, including: seven years of market exclusivity upon regulatory approval, if received; exemption from FDA application fees for Galactosemia; and tax credits for qualified clinical trials.

·Presented Phase 1/2 Data Highlighting Safety and Proof of Biological Activity for AT-001 in DbCM at The European Society for Cardiology (ESC) 6th World Congress in Athens, Greece. In May 2019, we presented two posters at ESC, the first of which was presented in the Late Breaking session and highlighted key data from a recently completed Phase 1/2 study in approximately 120 type 2 diabetic patients describing the safety, pharmacokinetics and proof of biological activity for AT-001 in DbCM. Supporting preclinical data from an animal model of DbCM was also presented, demonstrating that AT-001 prevents or reduces cardiac damage in a relevant disease model.

·Completed Initial Public Offering. In May 2019, we completed our IPO, generating approximately $34.6 million in net proceeds, after deducting underwriter discounts and commissions and offering expenses payable by us.

Financial Results

·Cash and cash equivalents totaled $41.1 million as of June 30, 2019, compared with $18.8 million at December 31, 2018.

·Research and development expenses for the three months ended June 30, 2019 were $4.3 million, compared to $1.9 million for the three months ended June 30, 2018. The increase of approximately $2.4 million was primarily related to costs associated with progressing our clinical trials, including an increase in clinical and pre-clinical expenses of $1.6 million and personnel expenses of $2.1 million due to the hiring of research and development personnel, including the Chief Medical Officer in August 2018. These increases are offset by a decrease in drug manufacturing and formulation expenses of $1.3 million.

·General and administrative expenses were $4.2 million for the three months ended June 30, 2019, compared to $0.4 million for the three months ended June 30, 2018. The increase of approximately $3.8 million was primarily related to personnel expenses of $2.2 million due to the increase in headcount, including the hiring of the interim Chief Financial Officer and the Controller, professional fees of $0.9 million due to increased legal and consulting fees, and other expenses of $0.7 million, primarily due to public relations efforts, travel expenses and recruiting efforts.

·Net loss for the second quarter of 2019 was $8.4 million, or $0.60 per basic and diluted common share, compared to a net loss of $3.2 million, or $0.58 per basic and diluted common share, for the second quarter of 2018.

Oncologie, Inc. Announces Clinical Trial Collaboration With Merck to Evaluate Bavituximab in Combination With KEYTRUDA® (Pembrolizumab) in Advanced Gastric or Gastroesophageal Cancer

On August 12, 2019 Oncologie, Inc., a clinical-stage biopharmaceutical company developing innovative oncology treatments targeting the tumor microenvironment, reported that it has entered into a clinical collaboration agreement with Merck (known as MSD outside the US and Canada) to evaluate the combination of Oncologie’s investigational drug Bavituximab, an antibody that blocks the activity of phosphatidylserine (PS), and Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in patients with advanced gastric or gastroesophageal cancer (Press release, Oncologie, AUG 12, 2019, View Source [SID1234538616]).

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Under the terms of the agreement, Oncologie will conduct a Phase 2 single arm open-label study to determine the efficacy and safety of Bavituximab in combination with KEYTRUDA in patients who have advanced gastric and gastroesophageal cancer, after they have failed at least one line of treatment. The study is expected to enroll approximately 80 patients in the U.S., United Kingdom, Korea and Taiwan. The study is anticipated to start enrollment in the second half of 2019.

"Gastric cancer is an area of significant unmet medical need in many parts of the world and we are committed to understanding the clinical benefit of Bavituximab and KEYTRUDA in this difficult-to-treat cancer," said Laura Benjamin, Ph.D., Founder and CEO of Oncologie. "This collaboration reflects a shared commitment to the goal of improving the lives of cancer patients in meaningful ways."

Keytruda is a registered trademark of Merck Sharp & Dohme Corp, a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About Bavituximab

Bavituximab is an investigational chimeric monoclonal antibody that targets the activity of phosphatidylserine (PS). Bavituximab is believed to reverse PS-mediated immunosuppression by blocking the engagement of PS with its receptors. PS-targeting antibodies have been shown to shift the functions of immune cells in tumors, resulting in multiple signs of immune activation and anti-tumor immune responses. This mechanism may play an important role in allowing other cancer therapies to more effectively attack tumors by reversing the immunosuppression that limits the impact of those treatments. Importantly, Bavituximab has also demonstrated a manageable safety and tolerability profile in previous clinical trials conducted to date, which may allow it to be combined effectively with other agents.

Emmaus Life Sciences Reports Sharply Improved 2019 Second Quarter Financial Results

On August 12, 2019 Emmaus Life Sciences, Inc. (Nasdaq: EMMA), a leader in sickle cell disease treatment, reported significantly improved financial results at its EMI Holding, Inc. (EMI) subsidiary for the 2019 second quarter and six-months ended June 30, 2019 (Press release, Emmaus Medical, AUG 12, 2019, View Source [SID1234538615]). EMI is the company’s principal operating subsidiary.

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2019 Second Quarter Financial Results of EMI
Net revenues of EMI for the 2019 second quarter increased 128% to $5.9 million, up from $2.6 million for the same period last year, and 11% from the first quarter of 2019. The increase was driven by the continuing roll-out and market acceptance of Endari, the first treatment approved by the FDA for sickle cell disease in nearly 20 years.

Total operating expenses equaled $6.3 million, compared with $5.1 million for the prior-year second quarter. The increase resulted primarily from higher selling costs related to the marketing and continued commercialization of Endari, higher research and development costs associated with EMI’s pilot/phase 1 diverticulosis study, and an increase in general and administrative expenses to support the commercialization of Endari and other business operations.

Operating loss for the 2019 second quarter was reduced substantially to $0.6 million, from $2.8 million last year.

"We have made substantial progress in the commercialization and roll-out of Endari, which is reflected in our 128% quarter-over-quarter revenue growth and improved financial results. Additionally, our recent merger has considerably strengthened our balance sheet and positioned Emmaus to better access the capital markets to support our growth," said Yutaka Niihara, M.D., M.P.H., Chairman and Chief Executive Officer of Emmaus. "We are continuing to broaden Endari’s expansion throughout the global marketplace, while studying the use of the same pharmaceutical-grade L-glutamine oral powder used in Endari as a new treatment option for patients with diverticulosis and diabetes."

2019 First-Half Financial Results
EMI’s net revenues for the first six months of 2019 increased 233% to $11.2 million, up from $3.4 million for the same period last year.

Total operating expenses were $12.0 million, compared with $10.2 million for the prior-year first half.

Operating loss for the six months ended June 30, 2019 was reduced to $1.2 million, versus $7.2 million last year.

As previously disclosed, in conjunction with and immediately prior to the merger, approximately $35.5 million principal amount of, and accrued interest on, outstanding convertible promissory notes and notes payable of EMI were converted into shares of EMI common stock and cancelled in the merger in exchange for Emmaus shares, with a resulting increase in stockholders’ equity. This conversion is expected to save Emmaus approximately $3.6 million in annual interest expense, which should benefit future cash flows. Additionally, in conjunction with the merger, EMI’s outstanding 10% senior secured debentures were amended and restated to extend their maturity date by six months to October 21, 2020 and to make the debentures convertible into common stock at a current conversion price of $9.52, subject to possible future adjustments.

Recent Highlights

Received clearance on its investigational new drug application from the Food and Drug Administration (FDA) for the study of a new L-glutamine treatment for patients suffering from diverticulosis. EMI commenced a pilot/phase 1 study of the safety and efficacy of its treatment at multiple study sites, with patents approved in the United States, the EU, China, Russia, Japan, South Korea, Mexico, Australia and Indonesia. Emmaus has patents pending related to diverticulosis treatment in Brazil and India.
Commenced a clinical study to determine the efficacy of the company’s pharmaceutical-grade L-glutamine in lowering blood sugar in patients with type II diabetes.
Signed an agreement with Express Scripts, one of the nation’s largest pharmacy benefits managers (PBM), and launched a commercial co-payment assistance program to help ensure that patients in need have access to Endari.
Entered into an exclusive agreement with taiba Healthcare for the registration, commercialization and distribution of Endari in certain countries throughout the Middle East and North Africa (MENA) region.
As previously reported, on July 17, 2019, Emmaus, formerly known as "MYnd Analytics, Inc.," completed its merger transaction with EMI, whereby EMI became a wholly owned subsidiary of Emmaus and the business of Emmaus became that of EMI. On July 18, 2019, Emmaus common stock began trading on The Nasdaq Capital Market under the symbol "EMMA."

Since the merger transaction occurred subsequent to the 2019 second quarter, Emmaus’ Quarterly Report on Form 10-Q for the quarter ended June 30, 2019 filed with the Securities and Exchange Commission reflects the historical business, assets, liabilities, financial condition and operations and financial results of the former MYnd Analytics which were spun off in conjunction with and prior to the merger. As a result, those results bear no relation to the company’s current business, assets, liabilities, financial condition or results of operations. EMI’s historical financial statements, along with pro forma financial information for Emmaus which give effect to the spin off and the merger, can be found in the exhibits to the Current Report on Form 8-K/A to be filed on August 14, 2019 which can be accessed at www.sec.gov.

Nasdaq Listing Status Update
Emmaus is currently reviewing a number of options related to maintaining the listing of its common stock on The Nasdaq Capital Market. The company has appealed the initial decision of the Listing Qualifications Staff of The Nasdaq Stock Market LLC. The appeal is scheduled to be heard on September 5, 2019. In the event the appeal is unsuccessful, Emmaus common stock may be eligible for quotation on the OTC Market.